UroGen Announces Completion of Enrollment in the Phase 3 UTOPIA Clinical Trial of UGN-103 for the Treatment of Recurrent Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer
UroGen Pharma (NASDAQ: URGN) has completed enrollment for its Phase 3 UTOPIA clinical trial of UGN-103, a next-generation treatment for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer. The trial enrolled 99 patients across multiple global centers.
UGN-103 utilizes UroGen's proprietary RTGel® technology, enabling sustained drug release and prolonged bladder exposure. The treatment aims to improve upon ZUSDURI™ with a shorter manufacturing process and simplified reconstitution. The trial's primary endpoint is complete response rate at three months, with responding patients followed up to 12 months for durability assessment.
The company secured patent protection for UGN-103 through December 2041 following a Notice of Allowance from the U.S. Patent and Trademark Office in September 2024.
UroGen Pharma (NASDAQ: URGN) ha completato l'arruolamento per il suo studio clinico di Fase 3 UTOPIA su UGN-103, un trattamento di nuova generazione per il carcinoma vescicale ricorrente a basso grado e rischio intermedio non muscolo-invasivo. Lo studio ha coinvolto 99 pazienti in diversi centri a livello globale.
UGN-103 sfrutta la tecnologia proprietaria RTGel® di UroGen, che permette un rilascio prolungato del farmaco e una maggiore esposizione della vescica. Il trattamento punta a migliorare ZUSDURI™ con un processo di produzione più rapido e una ricostituzione semplificata. L'endpoint primario dello studio è il tasso di risposta completa a tre mesi, con un monitoraggio fino a 12 mesi per valutare la durata della risposta nei pazienti che rispondono.
L'azienda ha ottenuto la protezione brevettuale per UGN-103 fino a dicembre 2041, dopo aver ricevuto un avviso di concessione dall'Ufficio Brevetti e Marchi degli Stati Uniti a settembre 2024.
UroGen Pharma (NASDAQ: URGN) ha completado la inscripción para su ensayo clínico de Fase 3 UTOPIA con UGN-103, un tratamiento de nueva generación para el cáncer de vejiga no músculo invasivo recurrente de bajo grado y riesgo intermedio. El ensayo incluyó a 99 pacientes en múltiples centros a nivel mundial.
UGN-103 utiliza la tecnología patentada RTGel® de UroGen, que permite una liberación sostenida del medicamento y una exposición prolongada en la vejiga. El tratamiento busca mejorar ZUSDURI™ con un proceso de fabricación más corto y una reconstitución simplificada. El objetivo principal del ensayo es la tasa de respuesta completa a los tres meses, con seguimiento de los pacientes respondedores hasta 12 meses para evaluar la durabilidad.
La compañía aseguró la protección de la patente para UGN-103 hasta diciembre de 2041 tras recibir un Aviso de Aprobación de la Oficina de Patentes y Marcas de EE.UU. en septiembre de 2024.
UroGen Pharma (NASDAQ: URGN)는 재발성 저등급 중간위험 비근육침습성 방광암 치료제인 UGN-103의 3상 UTOPIA 임상시험 등록을 완료했습니다. 이번 임상시험에는 전 세계 여러 센터에서 99명의 환자가 참여했습니다.
UGN-103은 UroGen의 독자적인 RTGel® 기술을 활용해 약물의 지속적인 방출과 방광 내 장기 노출을 가능하게 합니다. 이 치료법은 제조 과정을 단축하고 재구성 과정을 간소화하여 ZUSDURI™보다 개선된 효과를 목표로 합니다. 임상시험의 주요 목표는 3개월 시점에서 완전 반응률이며, 반응한 환자들은 내구성 평가를 위해 최대 12개월 동안 추적 관찰됩니다.
회사는 2024년 9월 미국 특허청으로부터 허가 통지를 받은 후 2041년 12월까지 UGN-103에 대한 특허 보호를 확보했습니다.
UroGen Pharma (NASDAQ : URGN) a terminé le recrutement pour son essai clinique de phase 3 UTOPIA portant sur UGN-103, un traitement de nouvelle génération pour le cancer de la vessie non invasif de bas grade à risque intermédiaire récurrent. L'essai a inclus 99 patients dans plusieurs centres mondiaux.
UGN-103 utilise la technologie propriétaire RTGel® d'UroGen, permettant une libération prolongée du médicament et une exposition prolongée de la vessie. Le traitement vise à améliorer ZUSDURI™ grâce à un processus de fabrication plus court et une reconstitution simplifiée. Le critère principal de l'essai est le taux de réponse complète à trois mois, les patients répondants étant suivis jusqu'à 12 mois pour évaluer la durabilité.
La société a obtenu la protection par brevet pour UGN-103 jusqu'en décembre 2041, suite à un avis d'acceptation délivré par l'Office des brevets et marques des États-Unis en septembre 2024.
UroGen Pharma (NASDAQ: URGN) hat die Rekrutierung für seine Phase-3-Studie UTOPIA mit UGN-103 abgeschlossen, einer neuartigen Behandlung für rezidivierenden niedriggradigen intermediären nicht-muskelinvasiven Blasenkrebs. Die Studie schloss 99 Patienten an mehreren globalen Standorten ein.
UGN-103 nutzt die firmeneigene RTGel®-Technologie von UroGen, die eine nachhaltige Wirkstofffreisetzung und verlängerte Blasenkontaktzeit ermöglicht. Die Behandlung zielt darauf ab, ZUSDURI™ mit einem kürzeren Herstellungsprozess und vereinfachter Rekonstitution zu verbessern. Das primäre Studienziel ist die komplette Ansprechrate nach drei Monaten, wobei ansprechende Patienten bis zu 12 Monate zur Dauerhaftigkeitsbewertung nachverfolgt werden.
Das Unternehmen sicherte sich den Patentschutz für UGN-103 bis Dezember 2041, nachdem es im September 2024 eine Zulassungsmitteilung vom US-Patent- und Markenamt erhalten hatte.
- Successful completion of Phase 3 trial enrollment with 99 patients
- Patent protection secured through December 2041
- Potential operational improvements over existing treatment ZUSDURI
- Proprietary RTGel® technology enabling sustained drug delivery
- Single-arm trial design may limit comparative efficacy data
- Results and efficacy data not yet available
Insights
UroGen's completion of Phase 3 enrollment for UGN-103 marks significant progress for their bladder cancer treatment pipeline with potential operational advantages.
UroGen has reached a critical milestone with the completion of enrollment in their Phase 3 UTOPIA trial for UGN-103, enrolling 99 patients across multiple global centers. This trial evaluates a next-generation formulation of mitomycin for treating recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).
The investigational UGN-103 leverages UroGen's proprietary RTGel® technology – a reverse-thermal hydrogel that enables sustained drug release and prolonged bladder exposure. This approach aims to improve upon their existing FDA-approved product ZUSDURI™ through a shorter manufacturing process and simplified reconstitution procedure, potentially offering operational advantages for healthcare providers while maintaining therapeutic benefits.
The single-arm trial design has patients receiving 75 mg of UGN-103 intravesically once weekly for six weeks, with complete response rate at three months as the primary endpoint. Responders will then enter a follow-up phase of up to 12 months to evaluate durability of response, which is critical for this type of recurring cancer.
The company has also secured intellectual property protection with a Notice of Allowance from the USPTO in September 2024, providing patent protection until December 2041 – a significant competitive advantage that secures their market position for nearly two decades if the treatment receives approval.
This development represents UroGen's strategic focus on expanding their uro-oncology portfolio beyond their first approved product, potentially streamlining treatment delivery while addressing an important medical need in bladder cancer management.
PRINCETON, N.J., July 07, 2025 (GLOBE NEWSWIRE) -- UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, today announced that it has completed patient enrollment in its Phase 3 UTOPIA clinical trial of UGN-103 (mitomycin) for intravesical solution, a next-generation formulation in development for the treatment of recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).
The UTOPIA trial enrolled 99 patients across multiple centers globally. UGN-103 uses UroGen’s proprietary sustained release RTGel® technology, a reverse-thermal hydrogel that enables sustained drug release and prolonged bladder exposure. UGN-103 is designed to offer improvements over ZUSDURI™ (mitomycin) for intravesical solution, including a shorter manufacturing process and simplified reconstitution procedure.
“Completing enrollment in the UTOPIA trial marks a significant milestone in our efforts to advance UGN-103 as a next-generation treatment option for patients with recurrent LG-IR-NMIBC,” said Michael Louie, M.D., EVP, Medical Affairs and Clinical Development, UroGen. “UGN-103 represents a pivotal advancement in our pipeline, aiming to deliver the same non-surgical benefits to patients as ZUSDURI, while also enhancing operational efficiency and convenience for providers.”
The UTOPIA trial is a single-arm, multicenter study evaluating the efficacy and safety of UGN-103. All enrolled patients receive 75 mg of UGN-103 via intravesical instillation once weekly for six weeks. The primary endpoint is complete response rate at three months, with responders entering a follow-up phase of up to 12 months to assess durability of response. For more information on the UTOPIA study, please visit clinicaltrials.gov/NCT06331299.
UroGen received a Notice of Allowance from the U.S. Patent and Trademark Office in September 2024 covering the use of UGN-103 for LG-IR-NMIBC, with patent protection expected through December 2041.
About UGN-103
In January 2024, UroGen entered into a licensing and supply agreement with medac to develop UGN-103 for LG-IR-NMIBC. UGN-103 is an innovative mitomycin formulation that aims to streamline manufacturing and reconstitution processes while providing intellectual property coverage until December 2041. It utilizes UroGen’s RTGel® technology for prolonged mitomycin exposure.
About ZUSDURI
ZUSDURI (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, approved for the treatment of adults with recurrent LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel® technology, a sustained release, hydrogel-based formulation, ZUSDURI is delivered directly into the bladder in an out-patient procedure by a trained healthcare professional using a urinary catheter to enable the treatment of tumors by non-surgical means. ZUSDURI is expected to be available in the U.S. on or around July 1, 2025. In the interim, patients can visit ZUSDURI.com for more information.
About Non-Muscle Invasive Bladder Cancer (NMIBC)
LG-IR-NMIBC affects around 82,000 people in the U.S. every year and of those, an estimated 59,000 are recurrent. Bladder cancer primarily affects older populations with increased risk of comorbidities, with the median age of diagnosis being 73 years. Guideline recommendations for the management of NMIBC include TURBT as the standard of care. Up to 70 percent of NMIBC patients experience at least one recurrence, and LG-IR-NMIBC patients are even more likely to recur and face repeated TURBT procedures. Learn more about non-muscle invasive bladder cancer at www.BladderCancerAnswers.com.
About UroGen Pharma Ltd.
UroGen is a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers because patients deserve better options. UroGen has developed RTGel reverse-thermal hydrogel, a proprietary sustained-release, hydrogel-based platform technology that has the potential to improve the therapeutic profiles of existing drugs. UroGen’s sustained release technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. Our first product to treat low-grade upper tract urothelial cancer and ZUSDURI (mitomycin) for intravesical solution are designed to ablate tumors by non-surgical means. UroGen is headquartered in Princeton, NJ with operations in Israel. Visit www.UroGen.com to learn more or follow us on X, @UroGenPharma.
About medac CDMO
The belief that health is humanity’s most valuable resource drives medac group. Since 1970, the mission of medac has been to improve patients’ quality of life worldwide by making the best medical treatments available. Since 2000, medac has been dedicated to improving patient outcomes globally by supporting pharmaceutical companies in bringing the best medical treatments to market. As a trusted Contract Development and Manufacturing Organization (CDMO), headquartered in Germany, medac CDMO is specialised in providing customised, high-quality services to customers and worldwide markets.
With a team of over 2,000 highly skilled professionals, medac CDMO offers comprehensive solutions tailored to the needs of clients worldwide. The cutting-edge facilities of medac group in Germany and the Czech Republic are equipped with the latest technologies to ensure precision, efficiency and compliance with the most stringent industry standards. From early-stage development to large-scale commercial production, medac CDMO is committed to foresight, progress, reliability and creative thinking which makes them a solution ahead.
The deep expertise, commitment to quality and flexible manufacturing capabilities enable medac CDMO to serve as a trusted partner for pharmaceutical and biotech companies looking to scale their operations and bring life-changing treatments to patients around the globe.
For more information, please visit www.medac-cdmo.com.
APPROVED USE FOR ZUSDURI
ZUSDURI (mitomycin) for intravesical solution is a prescription medicine used to treat adults with a type of cancer of the lining of the bladder called low-grade intermediate risk non-muscle invasive bladder cancer (LG-IR-NMIBC) after previously receiving bladder surgery to remove tumor that did not work or is no longer working.
IMPORTANT SAFETY INFORMATION
You should not receive ZUSDURI if you have a hole or tear (perforation) of your bladder or if you have had an allergic reaction to mitomycin or to any of the ingredients in ZUSDURI.
Before receiving ZUSDURI, tell your healthcare provider about all of your medical conditions, including if you:
- have kidney problems
- are pregnant or plan to become pregnant. ZUSDURI can harm your unborn baby. You should not become pregnant during treatment with ZUSDURI. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with ZUSDURI.
Females who are able to become pregnant: You should use effective birth control (contraception) during treatment with ZUSDURI and for 6 months after the last dose.
Males being treated with ZUSDURI: You should use effective birth control (contraception) during treatment with ZUSDURI and for 3 months after the last dose.
- are breastfeeding or plan to breastfeed. It is not known if ZUSDURI passes into your breast milk. Do not breastfeed during treatment with ZUSDURI and for 1 week after the last dose.
How will I receive ZUSDURI?
- You will receive your ZUSDURI dose from your healthcare provider 1 time a week for 6 weeks into your bladder through a tube called a urinary catheter. It is important that you receive all 6 doses of ZUSDURI according to your healthcare provider’s instructions.
- If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment.
- During treatment with ZUSDURI, your healthcare provider may tell you to take additional medicines or change how you take your current medicines.
After receiving ZUSDURI:
- ZUSDURI may cause your urine color to change to a violet to blue color. Avoid contact between your skin and urine for at least 24 hours.
- To urinate, males and females should sit on a toilet and flush the toilet several times after you use it. After going to the bathroom, wash your hands, your inner thighs, and genital area well with soap and water.
- Clothing that comes in contact with urine should be washed right away and washed separately from other clothing.
The most common side effects of ZUSDURI include: increased blood creatinine levels, increased blood potassium levels, trouble with urination, decreased red blood cell counts, increase in certain blood liver tests, increased or decreased white blood cell counts, urinary tract infection, and blood in your urine.
You are encouraged to report negative side effects of prescription drugs to the FDA.
Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to UroGen Pharma at 1-855-987-6436.
Please see ZUSDURI Full Prescribing Information, including the Patient Information, for additional information.
ZUSDURI™ is a trademark and UroGen® is a registered trademark of UroGen Pharma, Ltd.
Copyright©2025 UroGen Pharma, Inc. All rights reserved.
Forward-Looking Statements
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding: the belief that UGN-103, if approved, will offer several improvements over ZUSDURI, including a shorter manufacturing process and simplified reconstitution procedure; the potential opportunities for UGN-103 and benefits to patients and providers; UroGen’s pending intellectual property protection for UGN-103 potentially extending until December 2041; the expected timing for when ZUSDURI will be available in the U.S.; the estimated annual U.S. patient population and demographics for LG-IR-NMIBC; the potential of UroGen’s proprietary RTGel technology to improve therapeutic profiles of existing drugs other than mitomycin; and UroGen’s sustained release technology making local delivery potentially more effective as compared to other treatment options. Words such as “can,” “expect,” “may,” “plan,” “potential,” or other words that convey uncertainty of future events or outcomes are used to identify these forward-looking statements. These statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to: clinical results may not be indicative of results that may be observed in the future; potential safety and other complications related to UroGen’s products; risks related to our and our licensors’ ability to protect our respective patents and other intellectual property, including the fact that UroGen’s or medac’s pending patent applications may not be successful, and in such event, the duration of intellectual property protection would be more limited; the ability to maintain regulatory approval; complications associated with commercialization activities; labeling limitations; competition in UroGen’s industry; the scope, progress and expansion of developing and commercializing UroGen’s products and product candidates; the size and growth of the market(s) therefor and the rate and degree of market acceptance thereof vis-à-vis alternative therapies or procedures, such as surgery; UroGen’s ability to attract or retain key management, members of the board of directors and other personnel; UroGen’s RTGel technology and ZUSDURI may not perform as expected; new data relating to ZUSDURI, including from spontaneous adverse event reports and from the ongoing ENVISION trial, may result in changes to the product label and may adversely affect sales, or result in withdrawal of ZUSDURI from the market; the potential for payors to delay, limit or deny coverage for ZUSDURI; UroGen may not successfully develop and receive regulatory approval of any other product that incorporates RTGel technology; and the impacts of general macroeconomic and geopolitical conditions on UroGen’s business and financial position. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section of UroGen’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, filed with the SEC on May 12, 2025, the events and circumstances discussed in such forward-looking statements may not occur, and UroGen’s actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to UroGen as of the date of this release.
INVESTOR CONTACT:
Vincent Perrone
Senior Director, Investor Relations
vincent.perrone@UroGen.com
609-460-3588 ext. 1093
MEDIA CONTACT:
Cindy Romano
Director, Corporate Communications
cindy.romano@UroGen.com
609-460-3583 ext. 1083
FAQ
What is the primary endpoint of UroGen's Phase 3 UTOPIA trial for UGN-103?
How many patients were enrolled in UroGen's UTOPIA Phase 3 trial?
What advantages does UGN-103 offer compared to ZUSDURI?
How long is UroGen's patent protection for UGN-103?
What is the dosing regimen for UGN-103 in the UTOPIA trial?
